To find out whether these medications help patients with SMA, we have to conduct clinical trials. Here, we propose to prepare for clinical trials. We will invite SMA patients to join our research effort. We will examine them regularly to better understand their disease. The visits will include questions, physical exam, blood drawing, and sometimes X-rays and a skin biopsy. We will use modern computer methods to process the information. While we are doing this, we will plan a clinical trial. Once the clinical trial begins, we will offer SMA patients participation if they meet the criteria for that trial.